BioCryst Data Highlights Severe Psychosocial Burden of HAE on Pediatric Patients, Caregivers

BioCryst Pharmaceuticals Inc. (NASDAQ:BCRX) is one of the best upside stocks to buy now. On November 6, BioCryst Pharmaceuticals announced new data that showed the early and negative psychosocial burden of hereditary angioedema/HAE and the resulting emergency department/ED and hospital visits on pediatric patients and their caregivers.

Additionally, new one-year data from the ongoing APeX-P clinical trial showed an early and sustained reduction in monthly HAE attack rates in pediatric patients aged 2 to under 12 years treated with the once-daily oral granules of ORLADEYO (berotralstat). The second interim analysis of the APeX-P trial, which is the largest pediatric HAE prophylaxis trial, explored the one-year safety and effectiveness of ORLADEYO in children aged 2 to under 12 years, with a mean treatment exposure of 63.9 weeks.

The capsule formulation of ORLADEYO was previously approved by the FDA in December 2020 for HAE prophylaxis in patients aged 12 years and older. ORLADEYO is not recommended for the treatment of acute HAE attacks, and dosages over 150 mg once daily are not recommended due to the potential for QT prolongation. The most common adverse reactions are abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease.

BioCryst Pharmaceuticals Inc. (NASDAQ:BCRX) is a biotechnology company that develops oral small-molecule and injectable protein therapeutics to treat rare diseases.

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Disclosure: None. This article is originally published at Insider Monkey.